Reflecting on the STX209 Drug Trial

Well, the trial has ended.  And I STILL can't answer that 64 million dollar question: 

Were they on it, or weren't they?

We won't know what's been in these boxes until the end of the whole trial. When the drug is approved and ready for the market, we'll finally find out. So, estimates are, sometime in early 2014.  We won't know for around 2 1/2 years!

So now we start the extension.  The part where we give them the medication, beginning at a small dose and working our way up.

At our last appointment they staff did some testing to measure the boys' current cognitive abilities.  They were basically IQ tests. I hate IQ tests.  They don't measure what a person can do, they measure what a person can't do.  They are meant to separate the "averagely intelligent" from the "brilliant."  They don't work on the "horse of a different color."  (okay maybe not, but that's just how I feel.  My boys have tons of capabilities that those tests don't come near measuring.  I get a little testy when we talk about IQ tests.  Pun intended.)

But in this case, I welcomed the tests with wide open arms, because they expect the STX209 drug may help long term with cognitive abilities, and they want to have a clear view of where the boys are at right now, before they start the extension.  I also welcome it because I trust that the drug study will use methods of testing that will show more clearly what my boys are capable of, than a test that was created for "typical" children.

So I say, yes, measure away.

The doctors and assistants and staff at RUSH University are utterly wonderful.  They take such good care of us.  They make the process and procedures of a drug trial much less difficult and are always considerate of our special situation.  Probably because every one of their patients has a special situation.  They are quite familiar with those who are a "horse of a different color."

So now that the trial is over, I made a list of Pros and Cons to help me organize my thoughts on the experience.

PROS

• The boys have become excellent little travelers.  There have been lots of good experiences and benefits from the traveling aspect.
• I get to see people I mostly only know online at the clinic.  This last visit I saw Holly, and the visit before this one I saw Kristie (okay I barely saw Kristie, but Aliza saw her - that counts, right?)
• Like I mentioned above, the doctors and staff at RUSH are all fantastic, but it's especially great to be in a study with Dr. Elizabeth Berry-Kravis.  To be on her radar is to make sure you are in the right place at the right time, when the best treatments become available.
• Obviously, there is the benefit of helping a good medication get onto the market and available to people with autism and Fragile X.
• We are getting a head start on a medication that, long term, will help with mental abilities. By the time it's available through regular prescription, the boys will have already been on it for 2 years.

CONS

• The expense.  You can use your imagination and figure out what we've spent on hotels, gas, food, and incidentals on 5 trips to Chicago and back.  And oh, have I mentioned the toll roads?  Once you hit Illinois, there are 3 or 4 tolls to pay to drive there.  They range from 30 cents to $1.60, but starting in January they are raising rates to either $1.50 or $3!  Reimbursement is something we've talked about a lot, and heard lots of encouraging words, but we have yet to see any reimbursement.  We aren't doing this for the compensation, but we aren't independently wealthy either, and this is taking a huge toll on our family economy.
• Leaving Mark and Aliza back at home.  Splitting up the family so often is hard on all of us, but it's hardest on Aliza.  Her teacher told me this morning that Aliza mentioned she was missing me this week, and I found out she had a little stress breakdown in the cafeteria yesterday.  I know Mark does his best to make it easier for her.  But he's not The Momma.
• General hassle and inconvenience  - the time the boys have to take off school, and Mark has to take off work.

I guess this is one of those situations in life where I have remind myself to back up, and to see the whole forest.  I have to work to ignore each individual tree.  I've got to keep my eye on the prize - which would be, two boys who are intellectually able to be self-sufficient and happy.  That's what we hope this medication could do.  And if it does that, it's absolutely priceless.